Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation
Abstract Background Long-term effectiveness of asthma control medication has been shown in clinical trials but results from observational studies with children and adolescents are lacking. Marginal structural models estimated using targeted maximum likelihood methods are a novel statistiscal approac...
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| Format: | Article |
| Language: | English |
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BMC
2020-12-01
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| Series: | BMC Medical Research Methodology |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12874-020-01175-9 |
| _version_ | 1829473033772859392 |
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| author | Carolin Veit Ronald Herrera Gudrun Weinmayr Jon Genuneit Doris Windstetter Christian Vogelberg Erika von Mutius Dennis Nowak Katja Radon Jessica Gerlich Tobias Weinmann |
| author_facet | Carolin Veit Ronald Herrera Gudrun Weinmayr Jon Genuneit Doris Windstetter Christian Vogelberg Erika von Mutius Dennis Nowak Katja Radon Jessica Gerlich Tobias Weinmann |
| author_sort | Carolin Veit |
| collection | DOAJ |
| description | Abstract Background Long-term effectiveness of asthma control medication has been shown in clinical trials but results from observational studies with children and adolescents are lacking. Marginal structural models estimated using targeted maximum likelihood methods are a novel statistiscal approach for such studies as it allows to account for time-varying confounders and time-varying treatment. Therefore, we aimed to calculate the long-term risk of reporting asthma symptoms in relation to control medication use in a real-life setting from childhood to adulthood applying targeted maximum likelihood estimation. Methods In the prospective cohort study SOLAR (Study on Occupational Allergy Risks) we followed a German subsample of 121 asthmatic children (9–11 years old) of the ISAAC II cohort (International Study of Asthma and Allergies in Childhood) until the age of 19 to 24. We obtained self-reported questionnaire data on asthma control medication use at baseline (1995–1996) and first follow-up (2002–2003) as well as self-reported asthma symptoms at baseline, first and second follow-up (2007–2009). Three hypothetical treatment scenarios were defined: early sustained intervention, early unsustained intervention and no treatment at all. We performed longitudinal targeted maximum likelihood estimation combined with Super Learner algorithm to estimate the relative risk (RR) to report asthma symptoms at SOLAR I and SOLAR II in relation to the different hypothetical scenarios. Results A hypothetical intervention of early sustained treatment was associated with a statistically significant risk increment of asthma symptoms at second follow-up when compared to no treatment at all (RR: 1.51, 95% CI: 1.19–1.83) or early unsustained intervention (RR:1.38, 95% CI: 1.11–1.65). Conclusions While we could confirm the tagerted maximum likelihood estimation to be a usable and robust statistical tool, we did not observe a beneficial effect of asthma control medication on asthma symptoms. Because of potential due to the small sample size, lack of data on disease severity and reverse causation our results should, however, be interpreted with caution. |
| first_indexed | 2024-12-14T02:23:02Z |
| format | Article |
| id | doaj.art-f06b8e04390744a89376f13f6ed1fbb6 |
| institution | Directory Open Access Journal |
| issn | 1471-2288 |
| language | English |
| last_indexed | 2024-12-14T02:23:02Z |
| publishDate | 2020-12-01 |
| publisher | BMC |
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| series | BMC Medical Research Methodology |
| spelling | doaj.art-f06b8e04390744a89376f13f6ed1fbb62022-12-21T23:20:27ZengBMCBMC Medical Research Methodology1471-22882020-12-0120111010.1186/s12874-020-01175-9Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimationCarolin Veit0Ronald Herrera1Gudrun Weinmayr2Jon Genuneit3Doris Windstetter4Christian Vogelberg5Erika von Mutius6Dennis Nowak7Katja Radon8Jessica Gerlich9Tobias Weinmann10Institute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichInstitute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichInstitute of Epidemiology and Medical Biometry, Ulm UniversityInstitute of Epidemiology and Medical Biometry, Ulm UniversityInstitute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichPaediatric Department, University Hospital Carl Gustav Carus Dresden, TU DresdenComprehensive Pneumology Center CPC LMU Munich, member of the German Center for Lung Research (DZL)Institute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichInstitute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichInstitute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichInstitute and Clinic for Occupational, Social and Environmental Medicine, LMU University Hospital MunichAbstract Background Long-term effectiveness of asthma control medication has been shown in clinical trials but results from observational studies with children and adolescents are lacking. Marginal structural models estimated using targeted maximum likelihood methods are a novel statistiscal approach for such studies as it allows to account for time-varying confounders and time-varying treatment. Therefore, we aimed to calculate the long-term risk of reporting asthma symptoms in relation to control medication use in a real-life setting from childhood to adulthood applying targeted maximum likelihood estimation. Methods In the prospective cohort study SOLAR (Study on Occupational Allergy Risks) we followed a German subsample of 121 asthmatic children (9–11 years old) of the ISAAC II cohort (International Study of Asthma and Allergies in Childhood) until the age of 19 to 24. We obtained self-reported questionnaire data on asthma control medication use at baseline (1995–1996) and first follow-up (2002–2003) as well as self-reported asthma symptoms at baseline, first and second follow-up (2007–2009). Three hypothetical treatment scenarios were defined: early sustained intervention, early unsustained intervention and no treatment at all. We performed longitudinal targeted maximum likelihood estimation combined with Super Learner algorithm to estimate the relative risk (RR) to report asthma symptoms at SOLAR I and SOLAR II in relation to the different hypothetical scenarios. Results A hypothetical intervention of early sustained treatment was associated with a statistically significant risk increment of asthma symptoms at second follow-up when compared to no treatment at all (RR: 1.51, 95% CI: 1.19–1.83) or early unsustained intervention (RR:1.38, 95% CI: 1.11–1.65). Conclusions While we could confirm the tagerted maximum likelihood estimation to be a usable and robust statistical tool, we did not observe a beneficial effect of asthma control medication on asthma symptoms. Because of potential due to the small sample size, lack of data on disease severity and reverse causation our results should, however, be interpreted with caution.https://doi.org/10.1186/s12874-020-01175-9AdolescentsAsthmaChildrenControl medicationMarginal structural modelsTargeted-maximum likelihood estimation |
| spellingShingle | Carolin Veit Ronald Herrera Gudrun Weinmayr Jon Genuneit Doris Windstetter Christian Vogelberg Erika von Mutius Dennis Nowak Katja Radon Jessica Gerlich Tobias Weinmann Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation BMC Medical Research Methodology Adolescents Asthma Children Control medication Marginal structural models Targeted-maximum likelihood estimation |
| title | Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation |
| title_full | Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation |
| title_fullStr | Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation |
| title_full_unstemmed | Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation |
| title_short | Long-term effects of asthma medication on asthma symptoms: an application of the targeted maximum likelihood estimation |
| title_sort | long term effects of asthma medication on asthma symptoms an application of the targeted maximum likelihood estimation |
| topic | Adolescents Asthma Children Control medication Marginal structural models Targeted-maximum likelihood estimation |
| url | https://doi.org/10.1186/s12874-020-01175-9 |
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